To unlock more targeted, effective treatments, genetics researchers are exploring mutations responsible for some of the most common motor neuron diseases. These discoveries may lead to gene therapy treatment routes that do a better job of slowing disease progression than current medications. Much like gene therapy in oncology has brought improvement in cancer regression in recent years, we believe gene therapy—particularly therapy that targets and modulates RNA—is the future of neurology.
Run a more efficient pediatric clinical trial: Although pediatric trials overall cost roughly the same as adult clinical trials according to one analysis, they generally produce a more modest return on investment.
Movement disorders such as Parkinson’s disease, ataxias, tremor, dystonia, tics, Tourette’s, and dozens of rare diseases, […]
Less than a year ago, FDA approved the first cannabinoid-derived drug, Epidiolex. It’s a […]
“Microbiome” has become a consumer product buzzword, found in advertisements for soap and dozens […]
Neurological disorders and diseases affect all ages and ethnicities, and most result in long-term disability […]
In a statement last August, departing FDA Commissioner Scott Gottlieb outlined several steps the agency had […]
Advances in stroke management, the first cannabinoid-derived drug approved, new therapeutics for progressive multiple […]
Business as usual is not an option for conducting #clinicaltrials in the wake of the #COVID19 pandemic. Customized solutions and #patientcentricity are more optimal approaches in this environment. Jimmy El Hokayem, Ph.D., Associate Director of Program Development and Head of the Neurology and Regenerative Medicine Center of Excellence at Biorasi, shares his insights into #directtopatienttrials
Challenges in clinical trial recruitment are particularly prevalent in PD studies of disease modification, where the population of interest is difficult to find and study design is more complex. In fact, a recent PD clinical study showed that 55.6% of sites fell below projected recruitment goals.
Clinical trials in neurological diseases present a wide range of challenges and special considerations across the board. With the right experienced study team, a patient and disease pathobiology disease centered study process can be applied to mitigate many of those challenges.
Innovative and out of the box methods are often required for the recruitment of challenging populations […]